The treatment for butterfly children is safe for now. The ultimatum to close the Modena biotech Holostem falls

The treatment for butterfly children is safe for now.  The ultimatum to close the Modena biotech Holostem falls

Holostem is safe for the moment. The Modena biotechnology company that has developed a gene therapy to cure butterfly children’s disease will not close its doors tomorrow, as announced.

The Ministry of Infrastructure and Made in Italy (Mimit) led by Adolfo Urso saved the company at the last minute by signing the green light for the acquisition by the Enea Tech and Biomedical Foundation.

Enea Tech, supervised by Mimit, will therefore support Holostem with its budget from December 1st. The liquidation procedure is suspended for the moment. However, the life of biotech remains dependent on three conditions which Urso, in question time in parliament this afternoon, defined as mandatory. However, without clarifying what would happen in the event of non-compliance.

The first condition is “the immediate entry into Holostem of adequate and qualified management”. The second is “the development of an industrial plan within 6 months of the acquisition, which clearly highlights the strategies aimed at containing costs and increasing company turnover”.

This is a tricky point, because Holostem treats a rare genetic disease which in technical lexicon is called epidermolysis bullosa and which is unlikely to be profitable in the short term. The gene therapy developed in Modena has so far cured some patients on an experimental basis: therefore it does not even have a label with the precise price.

The costs for each patient treated are around hundreds of thousands of euros, even a million. However, at least from the results of the trials, they promise the lasting cancellation of a disease which in the most serious cases is lethal for children in the first years of life.

In addition to gene therapy, Holostem has developed a stem cell-based treatment for some forms of blindness dependent on the cornea which have instead completed trials, are authorized and used throughout Europe and have already found economic sustainability.

The third condition is “the company’s commitment to seeking private investors and industrial partners, finalizing their entry into the corporate structure within a suitable period of time from the acquisition and making use of the support of Invitalia in this regard”.

This is also a demanding condition, given that a private individual would hardly make a profit from Holostem. The precedent of Chiesi, a pharmaceutical company that supported butterfly children for several years, but then chose to back out, makes it clear that thinking in terms of profit may not be the right strategy.

In this respect, gene therapy for butterfly children is similar to other gene therapies for rare genetic diseases: they represent a great scientific success – and Italy, as demonstrated by the case of Modena, can cite several records – but certainly not a promise of I earn.

In one case (the treatment for the so-called bubble children) a non-profit like Telethon decided to shoulder all the costs, in order to save the lives of patients.

However, the mood in the Modena biotech today is one of joy. The petition to save Holostem, supported by the patients’ association Le Ali di Camilla, collected 60 thousand signatures in a few days, but the news of Urso’s signature came as a very welcome surprise. “These are the words that we all wanted to hear, because this treatment solution is important for many children” comments Stefania Bettinelli, president of Le Ali di Camilla.

The danger of immediate closure has been averted. But another period of economic negotiations opens for the company. If the cost of a human life could be budgeted, it would not be difficult to meet Urso’s three conditions. As long as only expenses and revenues are worth it, Holostem’s fate will still remain in the balance.


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